Identifying optimal dosage regimes under safety constraints: An application to long term opioid treatment of chronic pain

Statistics in Medicine
E B LaberSteve Ripa

Abstract

There is growing interest and investment in precision medicine as a means to provide the best possible health care. A treatment regime formalizes precision medicine as a sequence of decision rules, one per clinical intervention period, that specify if, when and how current treatment should be adjusted in response to a patient's evolving health status. It is standard to define a regime as optimal if, when applied to a population of interest, it maximizes the mean of some desirable clinical outcome, such as efficacy. However, in many clinical settings, a high-quality treatment regime must balance multiple competing outcomes; eg, when a high dose is associated with substantial symptom reduction but a greater risk of an adverse event. We consider the problem of estimating the most efficacious treatment regime subject to constraints on the risk of adverse events. We combine nonparametric Q-learning with policy-search to estimate a high-quality yet parsimonious treatment regime. This estimator applies to both observational and randomized data, as well as settings with variable, outcome-dependent follow-up, mixed treatment types, and multiple time points. This work is motivated by and framed in the context of dosing for chronic pain; ...Continue Reading

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Citations

Oct 6, 2020·Journal of the American Statistical Association·Qian GuanDipankar Bandyopadhyay
May 11, 2019·Annual Review of Statistics and Its Application·Michael R Kosorok, Eric B Laber

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